2016 At a Glance: What happened in Business, Finance and Innovations in Rare Disease Drug Discovery?

  • Alexion’s fresh rare diseases drug, Kanuma for treatment of babies with lysosomal acid lipase deficiency will undergo strict scanning by National Institute of Health and Care Excellence for its cost efficiency.

  • The pan-Canadian Pharmaceutical Alliance failed to settle the negotiation over drug price with Alexion pharmaceuticals for soliris treatment.
  • Marina Biotech entered into an evaluation and option agreement for the delivery of genome editing technology.
  • Pliant Therapeutics raised an amount of $45M in Series A financing from Third Rock Ventures, LCC for discovery and development of new techniques for treatment of fibrotic disorders.
  • An IRCM potential drug candidate, elamipretide, developed by stealth BioTherapeutics Inc. for treatment of primary mitochondrial myopathy procured Fast Track designation by FDA.
  • Catalyst pharmaceuticals’ Firdapse, a rare disease drug to treat Lambert-Eaton myasthenic syndrome met FDA speed breaker which demanded more supporting information by the company for approval.
  • BioCryst Pharmaceuticals’ drug, avoralstat, aimed to treat patients with hereditary angioedema failed in Phase 2 trials.
  • Auven and bellus are together set to complete the phase 3 confirmatory study of Kiacta, proposed drug for treatment of AA amyloidosis.
  • Researchers at the University of North Carolina and The Children’s Hospital of Philadelphia collaboratively developed a new gene therapy treatment for Factor VII deficiency, a rare blood disorder which could be treated by single injection.
  • Shire, a specialty pharma firm has settled upon buying Baxalta for around $32 billion to further expand its rare disease treatment reign.
  • Successful restoration of a rare metabolic disorder, propionic acidemia is possible, according to the collaborative study of HemoShear Therapeutics, Children’s National, MedStar Georgetown University Hospital. The study published in the Journal of Molecular Genetics and Metabolism.
  • Vtesse, Inc’s drug candidate VTS-270 for treatment of Niemann-Pick Type C1 Disease, which is in a pivotal Phase 2b/3 clinical trial got Breakthrough Therapy designation status by FDA.
  • Researchers got breakthrough in developing tests to diagnose two rare childhood diseases congenital disorders of glycosylation and metachromatic leukodystrophy. According to National Organization for Rare Disorders, the testing and treatment of these diseases were not known till now.


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